Neurodegeneration Exploring Commonalities Across Diseases: Workshop Summary (2013) / Chapter Skim
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1 Introduction
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Traditionally, research and development efforts for neurodegenerative diseases have primarily considered individual diseases separately, and largely separate research communities and patient advocacy groups have emerged. Recent findings, however, have revealed certain commonalities in 1 The planning committee's role was limited to planning the workshop, and the workshop summary has been prepared by the workshop rapporteurs as a factual summary of what occurred at the workshop.
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Looking across the neurodegenerative diseases, workshop presentations and discussions aimed to do the following: • Identify and discuss commonalities related to genetic and cellular mechanisms; • Identify areas of fundamental science needed to facilitate therapeu tics development; and • Explore areas of potential collaboration among the respective research communities and sponsors. Charge to Workshop Participants In her opening remarks, Story Landis, director of the National Institute of Neurological Disorders and Stroke and co-chair of the workshop planning committee, remarked that people who work on Alzheimer's disease and ALS, for example, typically have their own meetings and have few opportunities to "sit down, roll up their sleeves, and begin to talk about common mechanisms." This type of conversation, she said, could be extremely interesting and informative for participants working on these different diseases and could help advance understanding of potentially promising therapies.
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Planning committee members selected these four topics -- from among various potential candidates -- for discussion because of scientific interest in further exploring the mechanisms underlying these commonalities and/or the existence of promising therapeutics based on these mechanisms. Certain topics are well known to be shared mechanisms across many neurodegenerative diseases (e.g., protein aggregation)
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• Exploring commonalities across diseases may provide a promis ing approach: Workshop presentations and discussions highlighted many reasons to pursue an approach of examining commonalities across neurodegenerative diseases. Participants discussed genetic and pathological overlaps across multiple neurodegenerative dis eases, as well as genetic and cellular mechanisms that appear to be common across diseases, suggesting that cross-disease study could be appropriate and could help advance scientific understanding.
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Participants suggested various ways in which a cross-disease approach could help advance the development of therapeutics for neurodegenerative diseases. For example, one participant noted that identifying common threads across neurodegenerative diseases could help with target validation by at least showing that the result is based on multiple models rather than just one.
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o Sharing resources, tools, and data: In a variety of contexts, participants discussed the value of sharing resources, tools, and data among multiple investigators and/or academic and pharmaceutical entities. Examples of resources, tools, and data that could be shared included human genetics data to exam ine gene variants that may extend across disease populations, 4 Challenges and opportunities related to the use of animal models in research and development for nervous system diseases were explored in greater depth in a March 2012 workshop also hosted by the Institute of Medicine Forum on Neuroscience and Nervous System Disorders.
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o Funding: Several participants suggested various funding mechanisms that could help support work that examines and leverages commonalities across diseases. Ideas included the development of dedicated programs and funding to identify commonalities; support specifically aimed at identifying targets and therapies that may benefit more than one disease; and sup port for collaborations among scientists interested in advanc ing this type of approach.
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